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Immunotherapy Greatly Improves Survival for Infants with an Aggressive Form of Leukemia

These results mark a big step forward in the treatment of infants with this form of Acute Lymphoblastic Leukemia (ALL).

Rye Brook, N.Y., May 1, 2023 – Adding an immunotherapy drug to chemotherapy greatly improved survival for infants younger than 1 year with an aggressive form of acute lymphoblastic leukemia, according to a new study published in the New England Journal of Medicine. 

One-time infusion of blinatumomab was safe as initial therapy for newly diagnosed infants, increasing the chance of two-year disease-free survival (defined as time from treatment completion to disease recurrence or death from any cause) from 49% to 82%.

“Infants with this aggressive form of ALL, which is marked by an alteration in a gene called KMT2A, have a particularly poor prognosis, so this is a big step forward for improving the outcome of initial therapy,” said Gwen Nichols, M.D., chief medical officer of The Leukemia & Lymphoma Society. “Even with this big step forward, we need to keep advancing research because not all kids will benefit equally, and eventual relapses are possible.”

New targeted therapies for KMT2A-rearanged leukemias, including infant ALL and acute myeloblastic leukemia, are currently being investigated in clinical trials and will be part of the LLS-led Pediatric Acute Leukemia Master Clinical Trial (PedAL), which is matching children to therapies based on their unique tumor biology.

PedAL, a cornerstone of LLS’s Dare to Dream project, is expediting development of new targeted treatments for hard-to-treat childhood leukemias to reduce the need for one-size-fits-all chemotherapy that can be harsh in the short term and leave children with a lifetime of complications.

“The current results are a proof of concept for the type of targeted treatment we’re working toward with PedAL,” says Dr. Nichols. “The trial, which enrolled 30 patients across nine countries, also demonstrates the importance of the global collaboration we need to make a difference in pediatric cancers.”

The PedAL trial, the first of its kind led by a nonprofit, features a broad, global collaboration with industry leaders, academic institutions, and cutting-edge medical technology companies. Launched in 2022, PedAL is currently active in more than 160 screening sites with 30 treatment sites across the world.

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